Clinical manifestations before and during treatment with leniolisib
| Patient . | P1 . | P2 . | P3 . | P4 . | P5 . | P6 . | ||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Time point . | Before∗ (current status) . | During† (y of treatment, current status) . | Before (current status) . | During (y of treatment, current status) . | Before (current status) . | During (y of treatment, current status) . | Before (current status) . | During (y of treatment, current status) . | Before (current status) . | During (y of treatment, current status) . | Before (current status) . | During (y of treatment, current status) . |
| Infections‡ | CMV (RES) EBV (RES) Sinusitis (CON) Herpes simplex (CON) Parotitis (CON) Pulmonary (CON) | Otitis media acute (1; RES) Gastroenteritis (1; RES) Influenza A (1; RES) Cellulitis (1; RES) Cold sore (2; RES) HPV (5; RES) | Sinus (CON) Pneumonia (CON) | Upper respiratory viral, ×2 (2; RES) | CMV (RES) Parotitis (RES) EBV (CON) Otitis media (CON) Herpes oral (CON) Acute bronchitis (CON) Thrush oral (CON) Sinusitis (CON) Upper respiratory (CON) | Upper respiratory, ×3 (2, 5; RES) Stye on right eyelid (3; RES) Otitis externa (3; RES) Otitis media, ×2 (3, 4; RES) SARS-CoV-2 (5; RES) Pharyngeal sore (5; RES) Sinus, ×3 (4-5; RES) | Periorbital abscess (RES) Otitis media (CON) Sinus (CON) Pneumonia (CON) | Lyme disease (1; RES) | Otitis media (RES) Upper and lower respiratory (CON) Exudative pericarditis (Coxsackie virus) (RES) | Rhinitis (1; CON) Multiple relapses of respiratory (1, 2, 4, 5; CON) SARS-CoV-2 (5: RES) | Upper airway (CON) | Fungal skin on chest, back, and shoulders (1; RES) Multiple common colds (1, 4; RES) Folliculitis chin (3; RES) Otitis externa, ×3 (1, 2; RES) Influenza-like symptoms (1; RES) Otitis media (4; RES) SARS-CoV-2, ×2 (5, 6; RES) |
| GI involvement | Gastroparesis (1; RES) | Diarrhea (4; RES) | Protracted noninfectious diarrhea (CON) Chronic gastritis (CON) GI tract lymphoid hyperplasia (CON) | GI disorder§ (RES) Vomiting§ (RES) Worsening of diarrhea, ×2 (1; RES) Stomach ache (5; RES) | Gastroenteritis (1; RES) Diarrhea (1; RES) Reflux esophagitis, ×2 (2; CON) | |||||||
| Neurological involvement | Anxiety (CON) Migraines (CON) | Recurrent headaches (CON) | Recurrent headaches (CON) | Anxiety§ (CON) | White matter watershed zone defects (CON) | Facial nerve paresis (RES) | Anxiety (1; RES) Bilateral paresthesia of fingers (3; RES) | Headache (1; RES) Autism spectrum disorder (6; CON) | ||||
| Dermatological involvement | Keratosis pilaris on upper extremities (CON) Plantar warts (CON) Acne (CON) | Plantar warts (CON) | Nonpruritic rash on forearm and abdomen (1; RES) Seborrheic dermatitis (1; RES) | Seborrheic dermatitis (CON) | ||||||||
| Pulmonary conditions | Asthma (CON) Bronchiectasis (CON) | Asthma (CON) | Bronchiectasis (CON) Restrictive airway disease (CON) | Bronchiectasis (CON) Worsened productive cough (CON) Small airways disease with COPD features (CON) Lung lymphoid hyperplasia (RES) | COVID-19–associated mild dyspnea (5; CON) | Bronchial wall thickening (CON) | Tickling cough (2; RES) | |||||
| Malignancyǁ | Stage IV DLBCL (2010) | Hodgkin lymphoma with nodular sclerosis (2001) | MALT lymphoma (2005) | |||||||||
| Other | Aseptic meningitis (RES) | Hypertriglyceridemia (1; CON) | Wolff-Parkinson-White syndrome (CON) | Reactive arthritis (4; RES) | Hypertriglyceridemia (CON) Obesity (CON) Scoliosis (CON) | Failure to thrive (RES) | Hepatopathy (5; CON)¶ | |||||
| Patient . | P1 . | P2 . | P3 . | P4 . | P5 . | P6 . | ||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Time point . | Before∗ (current status) . | During† (y of treatment, current status) . | Before (current status) . | During (y of treatment, current status) . | Before (current status) . | During (y of treatment, current status) . | Before (current status) . | During (y of treatment, current status) . | Before (current status) . | During (y of treatment, current status) . | Before (current status) . | During (y of treatment, current status) . |
| Infections‡ | CMV (RES) EBV (RES) Sinusitis (CON) Herpes simplex (CON) Parotitis (CON) Pulmonary (CON) | Otitis media acute (1; RES) Gastroenteritis (1; RES) Influenza A (1; RES) Cellulitis (1; RES) Cold sore (2; RES) HPV (5; RES) | Sinus (CON) Pneumonia (CON) | Upper respiratory viral, ×2 (2; RES) | CMV (RES) Parotitis (RES) EBV (CON) Otitis media (CON) Herpes oral (CON) Acute bronchitis (CON) Thrush oral (CON) Sinusitis (CON) Upper respiratory (CON) | Upper respiratory, ×3 (2, 5; RES) Stye on right eyelid (3; RES) Otitis externa (3; RES) Otitis media, ×2 (3, 4; RES) SARS-CoV-2 (5; RES) Pharyngeal sore (5; RES) Sinus, ×3 (4-5; RES) | Periorbital abscess (RES) Otitis media (CON) Sinus (CON) Pneumonia (CON) | Lyme disease (1; RES) | Otitis media (RES) Upper and lower respiratory (CON) Exudative pericarditis (Coxsackie virus) (RES) | Rhinitis (1; CON) Multiple relapses of respiratory (1, 2, 4, 5; CON) SARS-CoV-2 (5: RES) | Upper airway (CON) | Fungal skin on chest, back, and shoulders (1; RES) Multiple common colds (1, 4; RES) Folliculitis chin (3; RES) Otitis externa, ×3 (1, 2; RES) Influenza-like symptoms (1; RES) Otitis media (4; RES) SARS-CoV-2, ×2 (5, 6; RES) |
| GI involvement | Gastroparesis (1; RES) | Diarrhea (4; RES) | Protracted noninfectious diarrhea (CON) Chronic gastritis (CON) GI tract lymphoid hyperplasia (CON) | GI disorder§ (RES) Vomiting§ (RES) Worsening of diarrhea, ×2 (1; RES) Stomach ache (5; RES) | Gastroenteritis (1; RES) Diarrhea (1; RES) Reflux esophagitis, ×2 (2; CON) | |||||||
| Neurological involvement | Anxiety (CON) Migraines (CON) | Recurrent headaches (CON) | Recurrent headaches (CON) | Anxiety§ (CON) | White matter watershed zone defects (CON) | Facial nerve paresis (RES) | Anxiety (1; RES) Bilateral paresthesia of fingers (3; RES) | Headache (1; RES) Autism spectrum disorder (6; CON) | ||||
| Dermatological involvement | Keratosis pilaris on upper extremities (CON) Plantar warts (CON) Acne (CON) | Plantar warts (CON) | Nonpruritic rash on forearm and abdomen (1; RES) Seborrheic dermatitis (1; RES) | Seborrheic dermatitis (CON) | ||||||||
| Pulmonary conditions | Asthma (CON) Bronchiectasis (CON) | Asthma (CON) | Bronchiectasis (CON) Restrictive airway disease (CON) | Bronchiectasis (CON) Worsened productive cough (CON) Small airways disease with COPD features (CON) Lung lymphoid hyperplasia (RES) | COVID-19–associated mild dyspnea (5; CON) | Bronchial wall thickening (CON) | Tickling cough (2; RES) | |||||
| Malignancyǁ | Stage IV DLBCL (2010) | Hodgkin lymphoma with nodular sclerosis (2001) | MALT lymphoma (2005) | |||||||||
| Other | Aseptic meningitis (RES) | Hypertriglyceridemia (1; CON) | Wolff-Parkinson-White syndrome (CON) | Reactive arthritis (4; RES) | Hypertriglyceridemia (CON) Obesity (CON) Scoliosis (CON) | Failure to thrive (RES) | Hepatopathy (5; CON)¶ | |||||
CMV, cytomegalovirus; CON, continuing; COPD, chronic obstructive pulmonary disease; DLBCL, diffuse large B-cell lymphoma; GI, gastrointestinal; HPV, human papillomavirus; MALT, mucosa-assisted lymphoid tissue; RES, resolved.
Manifestations that were reported before treatment (“Before”) were gathered from the OLE study relevant medical history and current medical conditions.
Manifestations that were reported during treatment were collected from the OLE study relevant medical history and current medical conditions, AE files, and by trial investigators. The relevant medical history and current medical conditions files contain manifestations that occurred during the DFT as well as the OLE study.
Chronic or recurrent infections are italicized. Infections and determination of status (resolved or continuing) were reported by trial investigators at their discretion.
Condition occurred after the DFT but before entry into the extension study.
All patients achieved full remission before entry into the trial, and there has been no recurrence of new lymphoma.
Hepatopathy was considered mild and stable at year 6.